News Specific Enzymes Cause Mutations That Fuel Aggressive Skin Cancer in Patients With RDEB, Study Shows

News Amryt’s AP101 for Treating EB Receives IND Clearance from FDA

News RDEB Is Main Suspect Behind SCC, a Rare Form of Skin Cancer, Study Shows

News 2 EB Research Groups Based Continents Apart Partner to Advance New Treatments

News Case Report Describes Successful Treatment of JEB with Terrasil Ointment

News Austrian Epidermolysis Bullosa Patient Inspires Dad to Establish World’s First ‘EB House’

News FDA Grants Fast Track Status to Castle Creek’s CCP-020 for Treatment of EBS

News Joint Venture Plans to Advance Potential EB Therapy RGN-137 into Phase 3 Trial

News Amryt’s AP101 Granted Rare Pediatric Disease Designation for EB

News Blocking Inflammation-linked PI3K-Delta Molecule Could Lead to EBA Therapy, Mouse Study Suggests

News Antibiotic Gentamicin May Benefit About 80% of Herlitz JEB Patients, Study Says

News Cord Blood Platelet Gel Holds Potential to Treat Epidermolysis Bullosa, Study Shows

News Debra’s Family-oriented EB Care Conference Kicks Off Sunday in Arizona

News Higher Levels of Granzyme B Play Key Role in EBA, Other Skin Blistering Diseases, Study Suggests

News Case Report Highlights Rare EBA Diagnosis in 21-Year-Old Patient

News Phase 1/2 Trial of DEB Therapy Candidate QR-313 Now Enrolling Patients, ProQR Announces

News Castle Creek Enrolls First Patient in Phase 1 Trial for CCP-020 as Treatment for EBS

News 2 New Mutations Found in Family with Epidermolysis Bullosa Pruriginosa, Case Study Reports

News Allogeneic Fibroblast Injections into Skin Lesions Are Safe, Effective for RDEB, Study Shows

News ProQR to Receive Up to $5 Million from Nonprofits to Develop QR-313 for DEB

Just Published, News Serlopitant’s Potential to Manage Itch in EB Patients Shown in Exploratory Trial

News Abeona Opens Manufacturing Plant to Support EB-101, and Other Gene and Cell Therapy Candidates

News US ‘Right to Try’ Law Meets with Mix of Praise and Criticism, Including Among Those with Rare Diseases

News EB Treatment Candidate BPM 31510 Granted Orphan Drug Designation in US

News #ECRD2018 – Genome Editing Might Be ‘Cure’ for Rare Diseases But Ethical Guidelines Needed, Panel Says

News #ERDC2018 – When Treating Rare Disease Patients, Don’t Overlook Quality of Life, Panel Urges

News FDA Grants Fast-Track Designation for Krystal Biotech’s Gene Therapy Candidate KB103 to Treat DEB

News Positive Interim Results of Phase 1/2 Trial Support FCX-007 Gene Therapy for RDEB

News Trial Shows Abeona’s Gene Therapy EB-101 Helps Wounds Heal in RDEB Patients

News Diacerein Ointment Granted Rare Pediatric Disease Status for EB Treatment

News #ECRD2018 – EU Must Do More for Rare Disease Patients, Eurordis Leaders Say

News ‘Rare Impact Awards’ Dinner Marks Orphan Drug Act and NORD at 35

News Rare Case of Genetic and Autoimmune Subtypes of EB Described in Report

News Krystal Biotech Doses First Patient in Phase 1/2 Trial Testing Gene Therapy Candidate KB103 for DEB

News Collagen Vesicles Released by Bone Marrow Stem Cells May Explain Their Therapeutic Potential, Study Shows

News Epidermolysis Bullosa Can Contribute to Esophageal Stenosis, Case Study Finds

News #ECRD2018 – Patient Access and Collaboration Focus of Eurordis Meeting May 10-12

News FDA Green-lights Krystal’s KB103 Gene Therapy Trial for DEB Patients

News Human Genome Project Team at NIH Honored for Advancing Research into Genetic Diseases

News Krystal Biotech’s KB103 Receives Orphan Medicinal Product Designation in Europe

News $3.8M Defense Grant Awarded to Develop Stem Cell Therapies for EB, Wound Care

News NIH Agency Pioneers Collaborative Research into Rare Diseases

News Researchers Identify a Blood Biomarker for Epidermolysis Bullosa-associated Squamous Cell Carcinoma

News Scientists Reduce Blistering in EBA Mice by Honing In on the Immune System

News EBA Can Lead to Acute Renal Failure, Case Report Contends

News SYK Protein a Key Regulator of Inflammatory Response in EBA Mouse Model, Study Finds

News Krystal Files Application to Start Clinical Trial of Gene Therapy Candidate KB103

News Fibroblast Skin Injections Show Promise in Healing Wounds in Dystrophic Epidermolysis Bullosa Patients, Study Finds

News Amryt Pharma Advances Lead Candidate AP101 to Global Phase 3 Trial for EB

News Krystal Biotech to File IND Application for KB103 to Treat Dystrophic Epidermolysis Bullosa

News Genome Sequencing and Its Clinical Potential Focus of NYC Rare Disease Day Event

News ‘A Disease May Be Rare, Hope Should Never Be,’ Says Boy with SMARD at Connecticut Rare Disease Day Event

News Debra of America, PC Project Collaborate for Patient-focused FDA Meeting in April

News Rare Disease Groups Welcome FDA’s Embrace of ‘Real World’ Data in Clinical Trials

News Better Method of Stem Cell Generation Shows Potential for Treating Epidermolysis Bullosa

News US Lawmakers Urge Bipartisan Support for Rare Disease Research, Patients’ Needs

News TWI Biotech Launching Phase 2 Trial to Test AC-203 Diacerein Ointment in EB Patients

News Dad Who Lost Baby to EB Setting Out on Cross-country Bike Ride to Raise Awareness, Funds

News Retrophin, Horizon Donate $3M Each to Rare Disease Institute at Children’s National

News ProQR Supports Rare Disease Day 2018, Honors Late Pioneer Henri A. Termeer

News BioNews to Cover 3 Rare Disease Day Events, Including NIH Conference

News Patient Advocacy Groups Worldwide Plan Events to Mark Rare Disease Day, Feb. 28

News Bone-anchored Hearing Aid Successfully Implanted in DEB Patient, Case Report Shows

News DNA, Protein Analyses Improve Diagnosis of Unusual Epidermolysis Bullosa Cases, Study Shows

News Rare Disease Groups, Patients Differ on ‘Right to Try’ Bill Before US Congress

News Diacerein Cream Shows Promise in Reducing Blisters in EBS Patients, Trial Shows

News Study Finds New Gene Mutation Leading to Recessive Dystrophic Epidermolysis Bullosa

News FDA Allows Pediatric Enrollment in Phase 1/2 Trial of FCX-007 Gene Therapy for RDEB

News NORD Fundraiser Sets 7,000-Mile ‘Bike, Run or Walk’ Goal for 7,000 Rare Diseases

News Abeona’s Gene Therapy EB-101 Receives FDA’s Regenerative Medicine Advanced Therapy Designation

News Rare Hair Loss Condition Linked to PLEC Gene Mutation and EBS Subtype for Only 2nd Time

News Factors Beyond COL7A1 Mutations May Contribute to Dystrophic EB Forms, Study Suggests

News Krystal Biotech Issued Patent Related to KB103 for Dystrophic EB

News Phase 2 Trial Data Validate New Investigator’s Global Assessment Scale

News Bactroban-resistant Bacteria Commonly Found in Skin Wounds of EB Patients, Study Shows

News Collagen VII Protein Important in Immune Response to Bacteria in EB, Mouse Study Shows

News DEB Patients Have More Pain, But No More Anxiety and Depression, Than Healthy People, Study Shows

News Abeona Readies Phase 3 Trial of EB Gene Therapy That Patient Helped Make Possible – Interview with CEO Timothy Miller

News Mutation in JUP Gene Can Cause Lethal Congenital EB, Case Report Shows

News Feeding Tubes May Help EB Patients Maintain Weight, Improve Quality of Life, Review Study Suggests

News Orally Administered Calcitriol Reduced Inflammation in EB Mice

News Celebrities Design Alex and Ani Jewelry Collection to Benefit EB Medical Research

News 2 Gene Mutations Identified as Possible Cause of EBS in One Patient, Researchers Say

News European Medicines Agency Grants Orphan Drug Status to QR-313 for Dystrophic EB

News Teen Ambassador Living with Epidermolysis Bullosa Is ‘Pride of Britain’

News ‘You See a Miracle’: Interview with Scientists on Gene Therapy That Saved Boy Dying of EB

News Rare Disease Patient Groups Unite to Preserve Orphan Drug Tax Credit in US

News KB103 Investigational Therapy Receives Clearance from RAC Committee

News Transplants of Genetically Altered Skin Treats Boy with Epidermolysis Bullosa, Study Shows

News Potential Gene Therapy for Dystrophic EB, KB103, Named an Orphan Drug by FDA

News Krystal Biotech Receives $700K Equity-Based Award to Develop Gene Therapy for DEB

News BERG Receives Partners in Progress Award for Efforts in Treating Epidermolysis Bullosa

News Children’s National, NORD Partner to Create Rare Disease ‘Centers of Excellence’

News #NORDsummit – Alternatives to Large Placebo Trials, Grant Awards Among Ways FDA Supporting Rare Diseases, Chief Says

News #NORDsummit – Despite Criticism, Orphan Drug Act Is Working to Advance Needed Treatments, FDA Says

News New Mutations Account for Genetically Unsolved Cases of EBS, Researchers Say

News Abeona to Advance Gene Therapy Research at Commercial Manufacturing Facility in Ohio

News Austrian Researchers Develop New Formulation of Diacerein to Treat EBS

News Fibrocell Reports Positive Results of Phase 1/2 Trial of FCX-007 Gene Therapy for RDEB

News Efforts Ramping Up to Treat EB: Interview with DEBRA